Unrelated bone marrow transplant for congenital amegakaryocytic thrombocytopenia: Report of two cases and review of the literature

Frangoul H, Keates‐Baleeiro J, Calder C, Manes B, Crossno C, Castaneda VL, Domm J. Unrelated bone marrow transplant for congenital amegakaryocytic thrombocytopenia: Report of two cases and review of the literature.
Pediatr Transplantation 2010: 14:E42–E45. © 2009 John Wiley & Sons A/S. :  CAMT i...

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Published inPediatric transplantation Vol. 14; no. 4; pp. E42 - E45
Main Authors Frangoul, Haydar, Keates‐Baleeiro, Jennifer, Calder, Cassie, Manes, Becky, Crossno, Christine, Castaneda, Victoria L., Domm, Jennifer
Format Journal Article
LanguageEnglish
Published Oxford, UK Blackwell Publishing Ltd 01.06.2010
Subjects
Bone Marrow Transplantation - adverse effects
congenital amegakaryocytic thrombocytopenia
Female
Humans
Infant
Megakaryocytes
pediatrics
Thrombocytopenia - congenital
Thrombocytopenia - therapy
unrelated donor bone marrow transplantation
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Summary:Frangoul H, Keates‐Baleeiro J, Calder C, Manes B, Crossno C, Castaneda VL, Domm J. Unrelated bone marrow transplant for congenital amegakaryocytic thrombocytopenia: Report of two cases and review of the literature.
Pediatr Transplantation 2010: 14:E42–E45. © 2009 John Wiley & Sons A/S. :  CAMT is a very rare cause of thrombocytopenia in infants. Most of the patients will progress to marrow failure. Allogeneic stem cell transplant remains the only curative therapy. We present two patients with CAMT who underwent an unrelated donor bone marrow transplant, one after developing marrow failure and another early in the course of the disease. Both patients tolerated the transplant with minimal toxicity and durable engraftment. We also present a comprehensive review of the literature for unrelated donor transplant for this condition.
ISSN:1397-3142
1399-3046
DOI:10.1111/j.1399-3046.2009.01131.x